News

$7.5 million commitment establishes Program for Individualized CF Therapy

TORONTO, October 21, 2015 – SickKids Foundation is pleased to announce a newly formed partnership with Cystic Fibrosis Canada which sees the creation of the Program for Individualized Cystic Fibrosis (CF) Therapy. The partnership includes a multi-year commitment to raise $7.5 million in support of CF research conducted at the Peter Gilgan Centre for Research and Learning at SickKids. This generous support will help SickKids researchers accelerate the discovery of new treatments and determine the most effective, personalized therapies for CF patients.

Together, Cystic Fibrosis Canada and SickKids researchers are creating a national resource to rapidly develop effective, personalized treatments for every person living with CF. With more than two thousand different CF gene mutations, each patient is different. The Program for Individualized CF Therapy is the world’s first resource dedicated to the discovery of more effective, personalized treatments for CF.

Investments in this initiative will create the platform necessary to harness technological and scientific advances through:

Stem cell collection: Tissue samples from CF patients who reflect the diversity of genetic mutations within the Canadian population are required to build a database of stem cells.

Genetic analysis: Each patient’s genetics will help researchers understand the factors affecting an individual’s disease severity and response to therapy, and allow them to predict which therapies will work best for each patient;

Patient-specific pre-clinical trials: Researchers will be able to rapidly test new and existing drug compounds on samples from multiple patients at the same time, predicting individual responses to a drug’s effectiveness before it is tested on patients;

Clinical testing: This will allow patients who might benefit from a specific therapy to receive treatment as soon as possible and rapidly bring new drugs to market;

Collaboration and innovation: The program will act as a hub for global collaboration and enable the world’s most innovative ideas in CF to come to fruition

Cystic Fibrosis Canada and SickKids have driven a number of landmark discoveries in CF research, including identifying the gene responsible for CF in 1989. Through research supported by Cystic Fibrosis Canada, SickKids scientists were also the first to prove the disease is a defect of a chloride channel – the process by which salt is drawn to the surface of mucus membranes. These discoveries led to the development of Ivacaftor, the first drug that repairs the defective CF gene instead of relieving diseasesymptoms.

Ivacaftor has dramatically improved quality of life for approximately five per cent of people affected by CF. However, no similar treatment exists for the majority of people affected by CF. Great advances have been made in CF in the recent decades; however, more work is needed to ensure every CF patient can control their disease and live a long, healthy life. Researchers are aiming to build on discoveries made within the past five years, including the development of targeted treatments, to help extend the lives of people living with CF.

QUOTES:

“The work of Cystic Fibrosis Canada and SickKids is a testament to what can be accomplished when organizations collaborate to improve the lives of the thousands of Canadians affected by CF. Now, we are calling on the community to support this new Program and help SickKids researchers revolutionize the way CF is treated, changing the future for people affected by this disease.”- Ted Garrard, President and CEO, SickKids Foundation

“We’ve made tremendous progress in treating cystic fibrosis and many patients are now hopeful that CF will not define, or delimit, their lives. But with over two thousands known mutations, we still have work to do. With the successes we’ve seen over the years, no one should be left behind. This collaboration with SickKids will propel the field of personalized CF therapy and offer tremendous hope for ALL Canadians with cystic fibrosis.”- John Wallenburg, Chief Scientific Officer, Cystic Fibrosis Canada

“We have never been closer to being able to treat cystic fibrosis as a chronic, yet controllable illness with the development of new drugs that directly target the defects caused by mutation in the CF gene. It’s clear that not all CF patients will respond well to the same drug so we need to be able to identify those people who will respond well to new therapies as they are developed. We will soon be able to compare multiple drugs in lung cells grown from stem cells to determine whether potential therapy will work for a particular individual with CF. Furthermore, new technology is allowing scientists a better understanding of complex genetic factors that affect an individual’s response to treatment. Together, we will be positioned to provide precision medicine for CF patients, ensuring that each individual receives the best therapy based on knowledge gained from their genetic makeup and the behaviour of their own lung cells.”- Dr. Christine Bear, Senior Scientist, Molecular Structure & Function research program, SickKids

About the Program for Individualized Cystic Fibrosis Therapy

Drawing from leading programs and infrastructure in stem cells, genetics, and cystic fibrosis, SickKids is a leading centre for research into the genetic basis of CF and the use of stem cells to identify new treatments. Now, Cystic Fibrosis Canada and SickKids are bringing this work to the next level by establishing the Program for Individualized Cystic Fibrosis Therapy, a national resource dedicated to individualized CF drug discovery. With more than two thousand different CF gene mutations, each patient is different. The goal is to ensure every CF patient has access to the life-changing treatments that will work best for them, as quickly as possible.

About Cystic Fibrosis Canada

Cystic Fibrosis Canada is one of the world’s top three charitable organizations committed to finding a cure for cystic fibrosis and is an internationally-recognized leader in funding CF research, innovation, and clinical care. We invest more funding in life-saving CF research and care than any other nongovernmental agency in Canada. We funded Dr. Lap Chee Tsui and his international research team who found the gene responsible for cystic fibrosis in 1989, and continue to fund leading-edge research in the hope of finding a cure or control for cystic fibrosis. Since 1960, Cystic Fibrosis Canada has invested $226 million in leading research, care and advocacy, resulting in one of the world’s highest median survival rates for Canadians living with cystic fibrosis. For more information, visit cysticfibrosis.ca.

About SickKids Foundation

Established in 1972, SickKids Foundation raises funds on behalf of The Hospital for Sick Children (SickKids) and is the largest charitable funder of child health research, learning and care in Canada. Philanthropy is a critical source of funding for SickKids – one of the world’s foremost paediatric healthcare institutions. Thanks to the generosity of the community, and as a result of a record-breaking year in fundraising and investment returns, SickKids Foundation generated an unprecedented $136 million for the fiscal year ending March 31, 2015. For more information, please visit sickkidsfoundation.com.

-30-

For more information, including an infographic with key details, photos, or to set up interviews with spokespeople, doctors, researchers or patient families, please contact:

Award-winning photographer and graphic designer Ian Pettigrew knew something wasn’t quite right when he was unable to have children. He was 38 years old when he discovered the real culprit behind his health issues – cystic fibrosis.